Over the next 2-2.5 years, biotech spin-out company Augustine Therapeutics will be working to understand both the underlying biology of Charcot-Marie-Tooth disease (CMT) and potential therapeutic options for patients. According to Charcot-Marie-Tooth News, the company recently received €1.2 million ($1.4m) in research funding from the Flanders Agency for Innovation and Entrepreneurship (VLAIO).
Augustine Therapeutics
On the company’s website, Augustine Therapeutics describes itself as a biotechnology company which:
focuses on the discovery and development of innovative medicines to benefit patients suffering from Charcot-Marie-Tooth disease (CMT) and other neuromuscular diseases. The newly formed company is rooted in the ground-breaking research at VIB and KU Leuven and supported by V-Bio Ventures, PMV, Advent France Biotechnology and Gemma Frisius Fund.
In this burgeoning research project, researchers will evaluate histone deacetylase 6 (HDAC6) inhibitors for patients with CMT. HDAC6 is deacetylating, meaning it removes acetyl groups from proteins. Through prior studies, researchers recognize that blocking HDAC6 can improve symptoms and quality of life (QOL) in multiple forms of CMT. However, researchers are still unclear exactly how these therapies work on a cellular level.
By better understanding both the underlying mechanisms of CMT, and how HDAC6 inhibition benefits patients, researchers from Augustine Therapeutics hope to potentially identify new treatment options moving forward.
Charcot-Marie-Tooth Disease (CMT)
Caused by a variety of gene mutations, Charcot-Marie-Tooth disease (CMT) is a rare inherited neurological disorder. It is sometimes referred to as hereditary motor and sensory neuropathy. Regardless of the genetic mutation, CMT is characterized by the degeneration of peripheral nerves. As these nerves are damaged, it prevents effective communication between nerves and muscles. Patients with CMT will often experience symptom onset in adolescence or early adulthood. However, in some more rare cases, symptoms may not develop until adulthood. Symptoms include:
- Hammertoes, high arches, and other foot deformities
- Muscle weakness in the hands, feet, and lower legs
- Frequent tripping
- Foot drop (difficulty lifting foot at the ankle)
- Decreased sensation
- Difficulty with walking and/or fine motor skills
- Joint stiffness
- Scoliosis (abnormal spinal curvature)
